SEC Filings

reversible
illness or perioperatively, as observed in the Finkel 2014 study, along with major developmental milestones for a healthy baby.

Existing Treatments for SMA

At this time, there is no FDA-approved treatment for SMA. The current standard of care for patients with SMA is limited to palliative
therapies, including life-long respiratory care, ventilator support, nutritional care, orthopedic care and physical therapy. Respiratory care is critical for patients with SMA Type 1 and
Type 2, as the cause of death in these subpopulations is usually respiratory-related.

Our Product Candidate: AVXS-101 for the Treatment of SMA Type 1

AVXS-101 is our proprietary gene therapy product candidate for the treatment of SMA Type 1. Because SMA is a neurodegenerative
disease, reduced levels of SMN protein lead to continued degeneration. The goal of AVXS-101 is to give patients a one-time treatment to restore the body's production of SMN protein to prevent further
degeneration. Based on preliminary observations of our ongoing Phase 1 clinical trial, we believe that AVXS-101 also enables increased motor function. AVXS-101 contains the four elements that
we believe are necessary for optimal delivery and function.